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Adult Stem-Cell Therapy Advances
BY Al Donner
Editor's note: This article has been updated from the print version.
Smiling broadly, 14-year-old Elizabeth Lobato steers her wheelchair a few doors from her home to a Baptist church in Riverside, Calif., where she volunteers to help preschoolers "because they really, really needed help."
A few weeks earlier, she guided her wheelchair into St. Peter’s Basilica.
Pope Francis gave her a blessing at a Vatican conference focusing on adult stem cells.
"It was such an amazing experience," she said. "Meeting the Pope was truly a special moment. He blessed me and my rosary."
Elizabeth was born with every bone broken in her tiny body, due to osteogenesis imperfecta, brittle bone disease.
As a child, her weak bones broke repeatedly, and her growth slowed, then halted entirely by age 6.
She was just 3-feet tall and very limited by her physical defect.
Today, Elizabeth is a happy, growing teen.
She is also a powerful example of the potential in adult stem-cell therapy advances.
Just a few years ago, Elizabeth was deteriorating.
On Easter morning 2010, she broke a leg, recalls her father, Terry Lobato: "We broke down; we were losing her!"
But Dr. Ed Horwitz, who was working with Elizabeth, was able to harvest rare adult stem cells from her father’s bone marrow.
Horwitz is a bone marrow and stem-cell researcher at Children’s Hospital of Philadelphia, specializing in stem-cell therapy.
He injected the stem cells into Elizabeth’s blood.
Soon, her bones began to strengthen, and growth resumed.
"The results were better than we ever dreamed of," said the physician.
All four children in his clinical trial experienced bone growth and bone strengthening. "It was a remarkable feat, never done before," he explained.
Elizabeth’s father, a middle-school science teacher, was ecstatic: "This was the miracle we were waiting for!"
Today, Elizabeth lives with optimism. She thinks the therapy will help other children with brittle bone disease, so that "they may never have to go through the pain I have."
Her future dream is "to work as a child life specialist at a hospital." Child life specialists help children cope with their hospital stays and explain medical procedures in kid-friendly ways, striving to eliminate the fear associated with medicine and doctors.
Regenerative therapy, based on stem cells, is seen by many as a new way to treat health problems as yet unsolved.
A decade ago, most of the talk was about embryonic stem cells (ESC). ESCs are undifferentiated cells, essentially blank slates, taken from four- or five-day-old fertilized eggs that are rapidly growing within a shell (blastocyst) the size of a dot. The shell contains 20-30 undifferentiated ESCs that soon will take on the specific roles in the growing human body.
Researchers thought ESCs could be redirected to take on specific roles for fighting human defects and illnesses. Taking ESCs from the blastocyst gives researchers blank cells that they can try to reprogram.
But that also ends the embryonic human life that is developing in the blastocyst.
That termination of new human life is the basis for the Church’s opposition to ESC research. The Church consistently holds that destroying human beings is immoral (see Catechism 2274-75).
That’s why the Lobatos — who have five other children, none with the bone disease that Elizabeth has — wanted Church-approved therapy like that which Horwitz could provide for Elizabeth.
"Elizabeth is our strength," said her father. "But as Catholics we could never ask anyone else to give up their life for our daughter."
A decade ago, the Church led a lonely, rudely dismissed fight against research using ESCs.
Rarely mentioned were adult stem cells (ASC), which are cells that have a specific function in the body (and are found in all humans, children as well as adults).
Research today uses ASCs in two ways. ASCs are used in therapy for their original purpose, which is the case with Elizabeth. Sometimes ASCs are given other tasks after being reprogrammed as pluripotent cells.
ASCs always have been a useful area of research, but they took a back seat to the dream envisioned for ESCs.
But in the last decade, that pattern shifted, and, with little public awareness, ASCs have become the primary focus of stem-cell research.
Major breakthroughs occurred in 2007, when scientists Shinya Yamanaka and Sir John Gurdon separately announced that they had discovered how to turn ASCs into pluripotent stem cells.
Gurdon and Yamanaka were awarded the 2012 Nobel Prize for Physiology or Medicine for unlocking how adult cells "can be reprogrammed to become immature cells capable of developing into all tissues of the body. Their findings have revolutionised (sic) our understanding of how cells and organisms develop."
Immensely complex, stem-cell research has not produced the quick results predicted a decade ago.
But it is beginning to show tangible success.
Current stem-cell therapeutic research areas include Alzheimer’s, diabetes, macular degeneration, stroke, leukemia, heart disease, osteoporosis, cartilage damage, even aging itself, and much more.
In recent weeks, researchers have publicized breakthroughs using adult stem cells in generating liver "buds" in mice that might be able to regenerate entire livers and in developing live "pulp" to rejuvenate otherwise dead teeth that now are removed by root-canal operations. The liver experiment showed initial success in crossing one substantial stem-cell hurdle, that of enabling new organisms to fully connect into a body's vascular system.
The University of Pittsburgh’s Dr. Stephen Badylak praised the liver research, but cautions that “our attempts at re-creating tissue structure are really very naive compared to what Mother Nature can do.”
Today, almost all of the clinical trials — the stage where lab-research results are tested on humans — under way are based on ASCs, according to the National Institutes of Health database.
In the U.S., more than 2,600 clinical trials are based on ASCs, but less than 1% — only eight — use ESCs.
Around the world, including the U.S., NIH identifies 4,500 clinical trials involving ASCs and only two dozen using ESCs.
Consequently, most federal stem-cell research funding today goes to ASC projects.
Yet, compared to the number of clinical trials, ESCs still receive a disproportionate share of research dollars. In 2012, ESC research received 22% of federal funding for stem-cell research.
Church and Science Support
Regardless of the funding discrepancy, adult stem cells are the future.
ESCs "lost their excitement," Horwitz says. "They are not as easy to work with as we once thought."
ESCs face both ethical and scientific challenges, among them the risk of introducing viruses or other unrelated defects into the recipient.
The shift in research is a little noted vindication for the Church’s position of strong support for ASC research and firm opposition to ESC therapy.
Much of the public perception was shaped a decade ago, when prominent entertainers, led by Michael J. Fox and the late Christopher Reeve, and much of the media and many political figures touted ESCs as the field with potential for new therapies.
The Church was overwhelmed in its effort to increase understanding that ESC research destroys lives. Ignored was its long support for research with ASCs because no harm is done to human beings.
The Church has continued to press for ASC research.
In 2011, then-Pope Benedict XVI said, "The potential benefits of adult stem-cell research are very considerable, since it opens up possibilities for healing chronic degenerative illnesses by repairing damaged tissue and restoring its capacity for regeneration."
This past April, the Vatican organized a conference, "Regenerative Medicine: A Fundamental Shift in Science & Culture," the one Elizabeth attended. It was a major step in restoring the Church’s role in ethical medical research, highlighting major advances in treating difficult diseases using ASCs. Participants exuded a sense of optimism and hope that important health benefits now appear possible and are beginning to be tested in human uses.
"We created this event so that we could educate the world on the ability of adult stem-cell therapies to address countless diseases and medical conditions, reducing suffering on a truly global scale," said Dr. Robin Smith, president of The Stem for Life Foundation and CEO of NeoStem, a stem-cell company supported by the Vatican.
At the conference, Nobel recipient Gurdon, an English development biologist, said, "I think it’s important for people who are not scientists to understand where work is in this field. I wish even more people were interested in where science is, rather than people who spend their time listening to celebrities on television.
"The kind of adverse feelings that some people have, I think, are entirely due to misunderstanding. They just don’t know enough about what the field is trying to do."
Elizabeth was one of several ASC beneficiaries recognized at the conference.
If the current research pattern continues, most of the hoped-for therapy will emerge from ASCs, therapy that is not lethal to an embryonic person — and much applauded by the Church.
And the Lobato family.
As Terry Lobato said of how adult stem-cell research has impacted his daughter’s life, "She loves the fact that she is growing. She’s loving life."
Al Donner writes from California.